Clinical Trial: A Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Brief Summary:

The purpose of this study in patients with mucopolysaccharidosis type II (MPS II) is below,

  • to collect the safety information of JR-141
  • to evaluate the plasma pharmacokinetics of JR-141
  • to explore the efficacy of JR-141 on MPS II-related central nervous system symptoms and general symptoms

Detailed Summary:
Sponsor: JCR Pharmaceuticals Co., Ltd.

Current Primary Outcome: Number of participants with Adverse Events [ Time Frame: 4 weeks ]

  • Adverse events
  • Laboratory tests
  • Vital signs
  • 12-lead electrocardiogram
  • Antibody
  • Infusion associated reaction


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]] [ Time Frame: 4 weeks ]
    Plasma concentration of JR-141
  • Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]] [ Time Frame: 4 weeks ]
    Plasma concentration of JR-141
  • Urinary and serum heparan sulfate (HS) /dermatan sulfate (DS) [ Time Frame: 4 weeks ]
  • HS/DS in CSF [ Time Frame: 4 weeks ]
    JR-141 concentration in CSF
  • Urinary total GAG [ Time Frame: 4 weeks ]
  • Liver and spleen volumes [ Time Frame: 4 weeks ]
  • Cardiac function [ Time Frame: 4 weeks ]


Original Secondary Outcome: Same as current

Information By: JCR Pharmaceuticals Co., Ltd.

Dates:
Date Received: April 10, 2017
Date Started: March 30, 2017
Date Completion: December 2017
Last Updated: April 24, 2017
Last Verified: April 2017